MY197192A - Regulation of gene expression through aptamer-modulated polyadenylation - Google Patents

Regulation of gene expression through aptamer-modulated polyadenylation

Info

Publication number
MY197192A
MY197192A MYPI2018001376A MYPI2018001376A MY197192A MY 197192 A MY197192 A MY 197192A MY PI2018001376 A MYPI2018001376 A MY PI2018001376A MY PI2018001376 A MYPI2018001376 A MY PI2018001376A MY 197192 A MY197192 A MY 197192A
Authority
MY
Malaysia
Prior art keywords
aptamer
gene expression
polyadenylation
regulation
modulated
Prior art date
Application number
MYPI2018001376A
Inventor
Michael J Volles
Olivier F Danos
Xuecui Guo
Original Assignee
Meiragtx Uk Ii Ltd
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by Meiragtx Uk Ii Ltd filed Critical Meiragtx Uk Ii Ltd
Publication of MY197192A publication Critical patent/MY197192A/en

Links

Classifications

    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/0008—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • A61K48/0066—Manipulation of the nucleic acid to modify its expression pattern, e.g. enhance its duration of expression, achieved by the presence of particular introns in the delivered nucleic acid
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/115—Aptamers, i.e. nucleic acids binding a target molecule specifically and with high affinity without hybridising therewith ; Nucleic acids binding to non-nucleic acids, e.g. aptamers
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86—Viral vectors
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/16—Aptamers
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/42—Vector systems having a special element relevant for transcription being an intron or intervening sequence for splicing and/or stability of RNA

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Biotechnology (AREA)
  • Biomedical Technology (AREA)
  • Molecular Biology (AREA)
  • Organic Chemistry (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Biochemistry (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Animal Behavior & Ethology (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Epidemiology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Medicinal Chemistry (AREA)
  • Virology (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Developing Agents For Electrophotography (AREA)

Abstract

The invention provides polynucleotide constructs for the regulation of gene expression by aptamer-based modulation of U1 snRNP-mediated suppression of polyadenylation and methods of using the constructs to regulate gene expression in response to the presence or absence of a ligand that binds the aptamer. The polynucleotide construct contains a U1 binding site in the context of a riboswitch comprising an effector region and an aptamer such that when the aptamer binds a ligand, target gene expression occurs. [No suitable figure]
MYPI2018001376A 2016-02-02 2017-02-02 Regulation of gene expression through aptamer-modulated polyadenylation MY197192A (en)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201662290200P 2016-02-02 2016-02-02
PCT/US2017/016279 WO2017136591A1 (en) 2016-02-02 2017-02-02 Regulation of gene expression through aptamer-modulated polyadenylation

Publications (1)

Publication Number Publication Date
MY197192A true MY197192A (en) 2023-05-31

Family

ID=59500159

Family Applications (1)

Application Number Title Priority Date Filing Date
MYPI2018001376A MY197192A (en) 2016-02-02 2017-02-02 Regulation of gene expression through aptamer-modulated polyadenylation

Country Status (14)

Country Link
US (2) US11697815B2 (en)
EP (1) EP3411081B1 (en)
JP (1) JP7184649B2 (en)
KR (1) KR102856245B1 (en)
CN (1) CN109511259B (en)
AU (1) AU2017213835C1 (en)
EA (1) EA201891721A1 (en)
ES (1) ES3059720T3 (en)
IL (1) IL260840B2 (en)
MX (1) MX2018009370A (en)
MY (1) MY197192A (en)
PH (1) PH12018501650B1 (en)
SG (1) SG11201806605RA (en)
WO (1) WO2017136591A1 (en)

Families Citing this family (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
KR102904879B1 (en) * 2015-11-12 2025-12-30 베이롤 칼리지 오브 메드신 Exogenous control of mammalian gene expression through aptamer-mediated modulation of polyadenylation.
BR112022019284A2 (en) 2020-03-24 2023-02-14 Meiragtx Uk Ii Ltd APTAMERS BINDING THIAMINE ANALOGS AND DERIVATIVES
EP4179112A4 (en) 2020-07-13 2024-11-20 The Board of Trustees of the Leland Stanford Junior University SYSTEMS AND METHODS FOR ASSESSING RNA STABILITY
EP4204015A4 (en) * 2020-08-31 2025-01-01 The Board of Trustees of the Leland Stanford Junior University SYSTEMS AND METHODS FOR THE PRODUCTION OF RNA CONSTRUCTS WITH ENHANCED TRANSLATION AND STABILITY
KR20240133712A (en) 2021-12-15 2024-09-04 메이라지티엑스 유케이 Ii 리미티드 Polycistronic expression of intestinal peptides
AU2024305450A1 (en) 2023-06-14 2026-01-22 MeiraGTx Gene Regulation Limited Small molecule ligands and aptamers

Family Cites Families (10)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US6692910B2 (en) 1997-07-28 2004-02-17 The University Of Connecticut Inhibition of a target messenger RNA with a modified U1 small nuclear RNA
US7563601B1 (en) 2005-06-01 2009-07-21 City Of Hope Artificial riboswitch for controlling pre-mRNA splicing
GB0701722D0 (en) * 2007-01-30 2007-03-07 Scottish Crop Res Inst Inhibition of gene expression
US9441221B2 (en) 2007-03-30 2016-09-13 Rutgers, The State University Of New Jersey Compositions and methods for gene silencing
EP2142672B1 (en) 2007-03-30 2012-09-05 Rutgers, The State University of New Jersey Compositions and methods for gene silencing
WO2010132665A1 (en) * 2009-05-15 2010-11-18 Yale University Gemm riboswitches, structure-based compound design with gemm riboswitches, and methods and compositions for use of and with gemm riboswitches
US9125931B2 (en) 2010-04-06 2015-09-08 Massachusetts Institute Of Technology Post-transcriptional regulation of RNA-related processes using encoded protein-binding RNA aptamers
EP2866822B1 (en) * 2012-07-05 2019-12-25 The Trustees Of The University Of Pennsylvania U1 snrnp regulates gene expression and modulates oncogenicity
KR102699584B1 (en) 2015-02-02 2024-08-28 메이라지티엑스 유케이 Ii 리미티드 Control of gene expression by aptamer-mediated regulation of alternative splicing
EP3494229B1 (en) 2016-08-03 2023-10-25 Meiragtx UK II Limited High throughput cell-based screening for aptamers

Also Published As

Publication number Publication date
IL260840A (en) 2018-09-20
JP7184649B2 (en) 2022-12-06
US20240191239A1 (en) 2024-06-13
EP3411081A1 (en) 2018-12-12
EP3411081A4 (en) 2019-12-11
US11697815B2 (en) 2023-07-11
IL260840B2 (en) 2025-12-01
WO2017136591A1 (en) 2017-08-10
US12559757B2 (en) 2026-02-24
AU2017213835A1 (en) 2018-09-06
CN109511259A (en) 2019-03-22
AU2017213835C1 (en) 2024-04-18
EP3411081C0 (en) 2026-01-07
KR20180117630A (en) 2018-10-29
EP3411081B1 (en) 2026-01-07
CA3048620A1 (en) 2017-08-10
ES3059720T3 (en) 2026-03-23
EA201891721A1 (en) 2019-02-28
PH12018501650B1 (en) 2024-07-03
PH12018501650A1 (en) 2019-06-03
CN109511259B (en) 2023-10-20
SG11201806605RA (en) 2018-09-27
IL260840B1 (en) 2025-08-01
JP2019503204A (en) 2019-02-07
AU2017213835B2 (en) 2024-02-01
US20200017858A1 (en) 2020-01-16
NZ745492A (en) 2025-03-28
KR102856245B1 (en) 2025-09-05
MX2018009370A (en) 2018-11-12
BR112018015819A2 (en) 2019-01-02

Similar Documents

Publication Publication Date Title
MX2018003689A (en) Pd-1 binding proteins and methods of use thereof.
MY197192A (en) Regulation of gene expression through aptamer-modulated polyadenylation
MX2018016038A (en) Compounds and methods for modulating rna function.
PH12017502180A1 (en) Tau-binding antibodies
PH12018500711A1 (en) Anti-pd1 antibodies and methods of use
IL255589B (en) Compounds targeting proteins, compositions, methods, and uses thereof
MX2020011749A (en) Compositions and methods for modulating pkk expression.
EP4299595A3 (en) Compositions and methods related to engineered fc constructs
EP3604535A3 (en) Methods and compositions for weed control
MX2015008999A (en) Mk2 inhibitors and uses thereof.
MX2016003843A (en) Irak inhibitors and uses thereof.
MY193723A (en) Combination therapy of tumor-targeted il-2 variant immunocytokines and antibodies against human pd-l1
MX2017016169A (en) Anti-her2 antibodies and methods of use.
SG10201808940WA (en) Nox inhibitor and nfкb inhibitor containing methoxyflavone
MY193677A (en) Tau-binding antibodies
EA201890268A1 (en) HERBICID COMPOSITION, WHICH CONTAINS CINMETHYLINE AND DIMETENAMIDE
SG10201909716RA (en) Modified j-chain
HK1216859A1 (en) Irak inhibitors and uses thereof
PH12015501059A1 (en) Anti-hemagglutinin antibodies and methods of use
PH12018501651A1 (en) Regulation of gene expression via aptamer-mediated control of self-cleaving ribozymes
MX2021006626A (en) Genetic markers for predicting responsiveness to therapy with hdl-raising or hdl mimicking agent.
WO2014172608A3 (en) Parthenolide derivatives and their modulation of processes controlled by regulated translation
MX2018012797A (en) Erbb inhibitors and uses thereof.
HK1222848A1 (en) Pyrrolotriazines as alk inhibitors
EP3140429A4 (en) Methods and compositions for scd, crt, crt-d, or sca therapy identification and/or selection